GEMTUZUMAB OZOGAMICIN for Acute promyelocytic leukemia

Inclusion criteria

• {"criterion_text":"- Newly diagnosed APL confirmed by the presence of PML/RARα fusion gene\n- Age <18 years\n- Written informed consent by parents or legal guardians\n- If applicable, female participants must have a negative pregnancy test by beta-HCG dosing.\n- Patients of child-bearing or child-fathering potential must be willing to adapt their own conduct so as not to procreate during the study participation and must contact their physician to identify the most appropriate approach strategy for this purpose starting from the time of enrolment and for 3 months after receiving the last drug dose"}

Exclusion criteria

• {"criterion_text":"- Patients with a clinical diagnosis of APL but subsequently found to lack PML/RARα rearrangement should be withdrawn from the study and treated on an alternative protocol\n- Significant liver dysfunction (bilirubin serum levels >3 mg/dL, ALT/AST serum levels greater than 5 times the normal values)\n- Creatinine serum levels >2 times the normal value for age\n- Significant arrhythmias, ECG abnormalities (ECG abnormalities: Congenital long QT syndrome; History or presence of significant ventricular or atrial tachyarrhythmia; Clinically significant resting bradycardia (<50 beats per minute); QTc >450 msec documented during screening EKG), other cardiac contraindications (L-FEV <50% or LV-FS <28%)\n- Neuropathy\n- Concurrent active malignancy\n- Uncontrolled life-threatening infections\n- Pregnant or lactating female\n- Patients who had received alternative therapy (APL not initially suspected; ATRA and/or ATO not available)"}

Primary endpoints

• {"endpoint_text":"- Event-free survival (EFS). This cumulative endpoint includes: no achievement of hematological complete remission after induction therapy; no achievement of molecular remission after three consolidation courses (molecular resistance); relapse (hematological/molecular); death, including early death, at 2 years from diagnosis. We aim at reaching a 3-year EFS probability of 90% (95% CI: 84.1-95.9%) and 80% (95% CI: 72.1-87.9%) in SR and HR patients, respectively","definition_or_measurement_approach":"EFS defined as the composite of failure to achieve hematological CR after induction, failure to achieve molecular remission after three consolidation courses, hematological/molecular relapse, or death (including early death) assessed at 2 years from diagnosis; study aims reference 3-year EFS probabilities for SR and HR groups."}

Secondary endpoints

• {"endpoint_text":"- Rate of hematological CR after induction\n- Rate of early and aplastic death during induction\n- Overall survival (OS)\n- Cumulative incidence of either hematological and molecular relapse (CIR)\n- Incidence of hematological and non-hematological toxicity\n- Kinetics of MRD clearance\n- Rate of molecular remission after 3 consolidation cycles\n- Assessment of PML/RARα transcript level reduction during treatment\n- Toxicity - hematological and non-hematological\n- Supportive care requirements\n- Total hospitalization days during therapy and health economic impact","definition_or_measurement_approach":"Secondary endpoints include response rates (hematological CR), early/aplastic death rates during induction, overall survival, cumulative incidence of relapse (hematological and molecular), toxicity incidence (hematological and non-hematological), kinetics of minimal residual disease (MRD) clearance, molecular remission rate after 3 consolidation cycles, assessment of PML/RARα transcript level reduction during treatment, supportive care needs, and total hospitalization days and health economic impact. Specific measurement timings and definitions are those described in the protocol (e.g., MRD kinetics by molecular assays; CIR calculated cumulatively)."}

Czechia

Latest Decision Or Authorization Date

06-12-2024

Number Of Participants

3

Sweden

Latest Decision Or Authorization Date

05-12-2024

Number Of Participants

2

Netherlands

Latest Decision Or Authorization Date

09-12-2024

Number Of Participants

3

France

Latest Decision Or Authorization Date

06-12-2024

Number Of Participants

31

Italy

Latest Decision Or Authorization Date

21-01-2025

Number Of Participants

60

Primary sponsor

Full Name

Associazione Italiana Ematologia Oncologia Pediatrica

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Italy

Third parties

• {"country":"Netherlands","full_name":"Julius Clinical International B.V.","duties_or_roles":"1","organisation_type":"Pharmaceutical company"}
• {"country":"Sweden","full_name":"Oriola Sweden AB","duties_or_roles":"15: Pharmacy resource","organisation_type":"Pharmaceutical company"}
• {"country":"Italy","full_name":"Ospedale Pediatrico Bambino Gesu","duties_or_roles":"1,10,11,13,14,2,4,5,6,8","organisation_type":"Hospital/Clinic/Other health care facility"}